The overall goal of the training component is the development of successful, independent, clinical and translational investigators who focus on the study of rare diseases and are able to lead a multidisciplinary team. The training of the scholar will be integrated into the clinical and translational training of the home institution (e.g., the Clinical and Translational Sciences Institute at UCSF). We will support advanced postdoctoral or clinical fellows (who will be independent investigators within the next year), junior faculty, or established investigators who wish to develop or refocus their careers on clinical research in rare diseases. Aim: To recruit and train highly qualified investigators in rare disease research.